Clinical and molecular characteristics of 20 Chinese probands with Mucolipidosis type II and III alpha/beta

Abstract Background Mucolipidosis (ML) II and III alpha/beta are lysosomal disorders caused by mutations in the GNPTAB gene which encodes the alpha and beta subunits of the heterohexameric enzyme, N-acetylglucosamine-1-phosphotransferase. Method To explore the clinical and molecular characteristics...

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Bibliographic Details
Main Authors: Yuyu Feng, Yonglan Huang, Xiaoyuan Zhao, Huiying Sheng, Xueying Su, Xi Yin, Liu Li, Wen Zhang
Format: Article
Language:English
Published: BMC 2024-12-01
Series:BMC Pediatrics
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Online Access:https://doi.org/10.1186/s12887-024-05223-x
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